George Biju, Mathews Vikram, Viswabandya Auro, Lakshmi Kavitha M, Srivastava Alok, Chandy Mammen
Department of Haematology, Christian Medical College, Vellore, Tamil Nadu, India.
Pediatr Hematol Oncol. 2010 Mar;27(2):122-31. doi: 10.3109/08880010903540542.
The authors compared the outcome in 100 children (61 boys, 39 girls; median age of 10.1 +/- 3.4 years) with aplastic anemia who underwent either immunosuppressive therapy (IST; n = 70) or hematopoietic stem cell transplantation (HSCT; n = 30) between 1998 and 2007. Conditioning regimes for HSCT were a combination of either cyclophosphamide (Cy) with antilymphocyte globulin (ALG) or fludarabine (Flu) with Cy or busulfan (Bu) +/- antithymocyte globulin (ATG). Stem cell source was bone marrow in 20 and peripheral blood stem cells (PBSCs) in 10. Patients undergoing IST received either equine ALG or ATG in combination with steroids and cyclosporine. Primary engraftment was seen in 25 children (83.3%), with acute graft-versus-host disease (aGvHD) in 5 (16.6%). The day 100 transplant-related mortality (TRM) was 30% and at a median follow up of 36 months (range: 6-197), the overall and disease-free survival is 70%. Among children who received IST, 60 children received ALG while 10 received ATGAM. Responses were seen in 27 children (43.5%), which was complete (CR) in 12 and partial (PR) in 15. At a median follow up of 38 months (range: 1-84), the overall survival is 37.1%, with 81.4% survival among responders and <10% survival among non-responders. HSCT would be the treatment of choice in children with severe aplastic anemia who have a human leukocyte antigen (HLA)-matched related donor and is superior to IST in this series from India.
作者比较了1998年至2007年间100例再生障碍性贫血患儿（61例男孩，39例女孩；中位年龄10.1±3.4岁）的治疗结果，这些患儿接受了免疫抑制治疗（IST；n = 70）或造血干细胞移植（HSCT；n = 30）。HSCT的预处理方案为环磷酰胺（Cy）与抗淋巴细胞球蛋白（ALG）联合，或氟达拉滨（Flu）与Cy联合，或白消安（Bu）+/-抗胸腺细胞球蛋白（ATG）。干细胞来源为20例骨髓和10例外周血干细胞（PBSC）。接受IST的患者接受马ALG或ATG联合类固醇和环孢素治疗。25例患儿（83.3%）实现了初次植入，5例（16.6%）发生了急性移植物抗宿主病（aGvHD）。100天移植相关死亡率（TRM）为30%，中位随访36个月（范围：6 - 197个月）时，总生存率和无病生存率为70%。在接受IST的患儿中，60例接受了ALG，10例接受了抗胸腺细胞球蛋白（ATGAM）。27例患儿（43.5%）出现反应，其中12例完全缓解（CR），15例部分缓解（PR）。中位随访38个月（范围：1 - 84个月）时，总生存率为37.1%，反应者生存率为81.4%，无反应者生存率<10%。对于有人类白细胞抗原（HLA）匹配的相关供体的重型再生障碍性贫血患儿，HSCT是首选治疗方法，在印度的这个系列研究中，其优于IST。
